Cure Ayra

Meet Ayra
To know Ayra is to know pure divine joy. At 6 years old, she loves listening to music, enjoys basic play with us and loves when we read books to her. But behind her bright smile, our brave little girl is fighting a battle no child should have to face.

The Diagnosis: SPG51
Recently, our world was turned upside down when Ayra was diagnosed with Spastic Paraplegia 51 (SPG51). This is an ultra-rare genetic condition that affects the nervous system, leading to physical and developmental challenges. Because it is so rare, traditional pharmaceutical companies are not funding a cure.

The Hope: We Have a Path to a Cure
We refuse to give up. We have partnered with a dedicated, world-renowned scientific team at University of Texas Southwestern that is working on a Gene Therapy Treatment specifically designed to halt and cure SPG51. The science is real, and the path forward is clear.

However, moving this groundbreaking science from the lab (preclinical research) into actual clinical trials to treat Ayra will cost $4 million.

Why We Need You
Time is our biggest enemy. SPG51 is progressive, meaning we are in a race against the clock to fund this research before the condition takes more from Ayra.

We have partnered with the Rare Village Foundation, a registered 501(c)(3) nonprofit, to manage this mission. This means 100% of your donations are tax-deductible and will go directly toward funding the SPG51 gene therapy treatment.

How You Can Make an Impact Today:

• Donate Online: Every dollar brings us closer to the lab, closer to the clinical trials, and closer to a cure.

• Give Monthly: Monthly donations help us reliably fund ongoing research month after month.

• Wire Transfers: We gratefully accept direct wire transfers. Please email us at info@rarevillage.org or cureayra@gmail.com to receive our bank details. Please include "Cure Ayra" in the memo or transfer message. 

• Check for Corporate Matching:
     a. Ask your employer if they match charitable donations to 501(c)(3) organizations! You can use our Tax ID/EIN (83-4699994) to easily find the Rare Village Foundation in your company's matching gift portal. 
    b. When submitting, please look for the "Designation", "Memo", or "Special Instructions" text box and type exactly: "Cure Ayra". This ensures the funds go straight to her research team. Thank you so much!

• Donate by Check: Please make checks payable to "Rare Village Foundation" and write "Cure Ayra" in the memo line. Mail them to: 6808 Old Glory Ct., McKinney, TX 75071.

• Donate Stocks: We also accept donations of stock! Please reach out to us at info@rarevillage.org or cureayra@gmail.com to coordinate a transfer.

• Share Ayra’s Story: Please share this page with your friends, family, and networks. Awareness is just as vital as funding.

The Roadmap to a Cure: How the $4M Will Be Used
Developing a first-of-its-kind gene therapy requires rigorous science, world-class partners, and strict FDA compliance. We are incredibly proud to be collaborating with some of the top scientific institutions in the world. Here is the strategic roadmap we are funding to take this therapy from the lab to the clinic:

• Phase 1: Preclinical Research & Vector Design
Timeline: ~12-24 Months 
Estimated Cost: ~$500,000
Partners: UTSW - Gray Lab, MIT Broad Institute, Boston Children's Hospital - Def-Lab - Harvard Medical School, Apertura, NIH
Focus: Designing the optimal gene therapy vector, engineering blood-brain barrier delivery (TfR1 CapX), and testing efficacy in lab models.

• Phase 2: IND-Enabling & Toxicology Studies
Timeline: ~6-9 Months
Estimated Cost: ~$600,000
Partners: UTSW - Gray Lab, NIH
Focus: Mandatory FDA safety testing, large-model toxicology, and preparing the official Investigational New Drug (IND) application.

• Phase 3: Trial Preparation & Clinical Manufacturing (cGMP)
Timeline: ~12 Months
Estimated Cost: ~$2,150,000
Partners: Gene Therapy Manufacturing Company
Focus: Producing the ultra-pure, clinical-grade viral vector required by the FDA for human dosing.

• Phase 4: Clinical Trial
Timeline: ~24-48 Months
Estimated Cost: ~$750,000
Partners: UTSW - Gray Lab, Gene Therapy Manufacturing Company, Children's Hospital
Focus: First-in-human dosing, extensive patient monitoring, and trial execution for Ayra and the initial patient cohort.

Note: To accelerate our path to a cure, several of these phases will be run in parallel whenever scientifically and safely possible.

From the bottom of our hearts, thank you for standing with Ayra, for giving us hope, and for helping us fight for her future.